Mirum Pharmaceuticals presents long-term data from LIVMARLI studies

Mirum Pharmaceuticals announced that data from new analyses showing long-term treatment with LIVMARLI were presented during the European Association for the Study of the Liver Annual Congress, June 5-8, 2024, in Milan, Italy. Presentations from studies evaluating LIVMARLI oral solution in patients with Alagille syndrome and Progressive Familial Intrahepatic Cholestasis included: THU-094: Clinical Benefits with Maralixibat for Patients with Alagille Syndrome are Durable Through 7 Years of Treatment: Data from the MERGE Study: An analysis of ALGS studies evaluating LIVMARLI for up to seven years of treatment were presented to demonstrate the durability of response to LIVMARLI across multiple clinical and laboratory outcomes. Data demonstrated that nearly all patients who remained on LIVMARLI for seven years experienced benefit including clinically meaningful reductions in pruritus and serum bile acids, along with improvements in height. THU-129: Maralixibat Improves Growth in Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH/MARCH-ON Trials: This analysis reported on the long-term impact of LIVMARLI on improvements in growth across a variety of PFIC types in 60 patients from the All-PFIC cohort of the MARCH/MARCH-ON clinical trials. Patients treated with LIVMARLI from the All-PFIC cohort had a statistically significant improvement in height and weight z-scores which persisted through 82 weeks of treatment. THU-158: Improvements in Serum Bile Acid Levels are Associated with Improvements in Key Markers of Liver Health After Maralixibat Treatment in Children with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH/MARCH-ON Trials: This analysis aimed to assess correlations between serum bile acid improvements and key liver parameters, including bilirubin. These data showed that reductions in sBA after treatment with LIVMARLI are correlated with reductions in bilirubin, a key marker of liver disease. These results indicate the potential for LIVMARLI to have disease modifying effects and improvements in liver health for patients with PFIC.